FDA approves first therapy for recurrent respiratory papillomatosis

Published 15/08/2025, 12:14
FDA approves first therapy for recurrent respiratory papillomatosis

GERMANTOWN, Md. - The U.S. Food and Drug Administration has granted full approval to PAPZIMEOS (zopapogene imadenovec-drba) for the treatment of adults with recurrent respiratory papillomatosis (RRP), Precigen, Inc. (NASDAQ:PGEN) announced Friday. The news comes as the company’s stock has shown remarkable strength, posting a 73% gain over the past year and maintaining strong momentum with a 65% rise year-to-date, according to InvestingPro data.

PAPZIMEOS becomes the first and only FDA-approved therapy specifically targeting RRP, a rare disease affecting an estimated 27,000 adults in the United States. The condition is caused by chronic human papillomavirus (HPV) types 6 or 11 infection, resulting in recurrent benign tumors in the respiratory tract. For Precigen, currently valued at $551 million, this approval represents a significant commercial opportunity. InvestingPro analysis reveals that analysts anticipate strong sales growth for the company, with revenue projected to grow by 305% in the current fiscal year. Subscribers can access 8 additional key insights about Precigen’s financial health and growth prospects on InvestingPro.

The therapy, administered via four subcutaneous injections over a 12-week period, is designed to generate an immune response against HPV 6 and 11 proteins. The FDA granted full approval rather than accelerated approval, eliminating the need for a confirmatory clinical trial.

The approval was based on data from an open-label, single-arm pivotal study where 51% of patients (18 out of 35) achieved complete response, requiring no surgeries in the 12 months following treatment. Of those complete responders, 15 patients evaluated at 24 months demonstrated continued complete response.

"For more than a century, since RRP was first recognized as a distinct disease, patients have had to rely on repeated surgeries to manage this relentless condition," said Helen Sabzevari, President and CEO of Precigen, in the press release.

Prior to this approval, RRP management consisted primarily of repeated surgeries that did not address the root cause of the disease. These procedures can lead to significant morbidity and burden on both patients and the healthcare system. While the company maintains a healthy balance sheet with more cash than debt and strong liquidity ratios, InvestingPro’s comprehensive analysis indicates the stock is trading slightly above its Fair Value. Get detailed insights and access to the full Pro Research Report, available for over 1,400 US stocks, to make more informed investment decisions.

The most common side effects reported during clinical trials included injection site reactions, fatigue, chills, fever, muscle aches, and nausea.

Precigen has established a patient support program called Papzimeos SUPPORT to assist with insurance navigation and financial assistance. The company will host a conference call on Monday to provide additional details about the approval and commercialization plans.

In other recent news, Precigen Inc. has been the focus of several analyst reports and corporate developments. The company recently reported its financial results for the first quarter of 2025 and provided a business update, emphasizing its preparations for the potential launch of its therapeutic candidate PRGN-2012 in the second half of 2025. JMP Securities has maintained a Market Outperform rating and a $6 price target on Precigen, expressing optimism about the company’s commercial readiness. Cantor Fitzgerald also reiterated an Overweight rating, highlighting the company’s progress in securing insurance coverage for PRGN-2012, with expected pricing between $300,000 and $500,000.

Precigen is actively enrolling patients in a confirmatory trial for PRGN-2012, with a target of around 35 patients, following guidance from the U.S. Food and Drug Administration. The company’s commercial and manufacturing preparations are reportedly advancing, with medical liaisons engaging healthcare providers to raise awareness about the upcoming product. Both JMP and Cantor Fitzgerald have noted the strategic efforts Precigen is making to ensure a successful product launch. These developments underscore the company’s focus on addressing the needs of Recurrent Respiratory Papillomatosis patients.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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